4 Essential Drivers In Clinical Development For Emerging Biotechs

Enrollment, enrollment, enrollment has been the sponsor’s classic mantra for achieving success in a clinical trial. Rather, we should replace this notion with four equally essential building blocks: 1) the selection of the right CRO, 2) the identification of study sites, 3) the engagement of sites, and 4) the enrollment of patients. Clinical development teams must be laser-focused on all four by leveraging advanced technologies (high tech) and personal interactions (high touch).

Selecting The Right CRO

For emerging biotechs without in-house clinical operations, a critical first step is to identify an enduring, transparent, and trustworthy partner, a CRO able to provide skills and experience in:

• Therapeutic modality, such as biologics, gene, or cell therapies
• Disease target, including assessment of endpoints and biomarkers
• Innovative study designs, such as adaptive clinical trials
• Agile operational models and financial management
• Data analytics, digitalization, and remote monitoring
• Drug distribution and management
• Quality of processes and personnel
• Cost of services

Finding The Right Clinical Site

For biopharma organizations without an established network of principal investigators (PIs), sub-PIs and/or key opinion leaders (KOLs), it’s essential to identify this network and develop relationships based on:

• Scientific presentations (podiums) and publications in scholarly journals (impact factor)
• Clinical trial activity in targeted disease, including public (clinicaltrials.gov) and proprietary databases
• Access to patients in medical practice (claim databases)
• Grant funding and awards (professional recognition)
• Social media presence (digital opinion leaders)

To be able to cross-reference all these parameters and create an accurate profile of study sites and clinicians, it’s essential to either create or have access to a comprehensive database that can be interrogated. The sources for this database can be: 1) public, such as internet search, clinicaltrials.gov, social media; or 2) proprietary, such as claims data or recommendations from existing relationships and advisory boards.

While established biopharmaceutical organizations can develop and curate iteratively in-house databases, emerging biotechs need specialized vendors offering comprehensive access and bespoke technical support.

The criteria and “strategic lens” to query this database should focus on the opportunities or challenges to be addressed. The outcome will consist of identifying PIs/KOLs and study sites that can help validate the underlying science, amplifying awareness of clinical data through publications and potentially support a shift in a treatment paradigm. This approach also will help articulate the broader medical affairs and future commercial strategy.

This strategy also can identify rising stars — clinicians who are starting to receive recognition and status by leading trials, presenting at conferences, and publishing clinical trial results. They leverage multiple mediums to create and share content, thus establishing their presence, voice, and reach with a digital footprint. They are often early to communicate new information, leading conversations online and serving as trusted sources.

Engaging Client Sites, Especially Through DCTs

Once study sites have been identified and incorporated into the clinical program, we must assess: 1) the clinical site technology and infrastructure, such as electronic health record systems, data management and pharmacy capabilities; 2) the clinical site personnel, including investigators and coordinators; and 3) the patient-friendliness at the site. This means ensuring that the clinicians and site team can support the patient throughout the clinical trial journey.

To ensure full engagement of the clinical site, we must develop: 1) effective and structured communication channels; 2) a coordinated approach at the site, especially for the initial interactions, such as site activation and initiation visits; and 3) supportive documentation and tools to ensure the site becomes familiar with the management of the target disease, prescription, and practice patterns.

DCTs, also known as hybrid trials, remote trials, or direct-to-patient trials, where parts or all of the trial happen outside of a traditional trial site, depend on a seamless interaction of high tech with high touch. Technology cannot replace personal interactions with remotely monitored patients. Swift adoption of virtual interactions between clinicians and patients, such as telemedicine, wearables, home visits, and direct delivery of study drugs and materials to patients’ homes, should not replace real-life interactions.

Enrolling (And Engaging) The Patient With Lay Language

The statement that “the treatment has been developed with the patient in mind,” doesn’t equate to a trial being patient centric. Traditionally, the patient is rarely involved in the R&D process until late-stage trials. Modern biopharma organizations should involve patients early on by considering patients as research partners and involving them in the clinical development program.

The sponsor could develop patient-focused printed and digital materials, such as recorded interviews with KOLs and educational videos that use lay language to:

• Explain safety and efficacy aspects of the clinical trial
• Explain the therapy’s mechanism of action (MOA)
• Explain the importance of biomarkers, precision, and personalized medicine
• Obtain endorsement from IRBs and other ethics committees
• Obtain endorsement of advocacy groups
• Obtain endorsement from participating patients once they have consented

Furthermore, other aspects that can be relevant to patients are logistics for study visits at the hospital or at home, safe drug storage and delivery, and guidance for patient responsibilities, such as dosing diaries and PROs.

All these elements can be discussed with patient advisory boards or focus groups that can solidify learnings, optimize execution, and lead to a holistic integration of patients in clinical trials. Ideally, patients can provide genuine and candid feedback regarding protocol complexity and design of study procedures, potentially leading to iterative protocol revisions.

High tech and digitalization are entering the clinical development space at a dizzying speed. It may seem that buzzwords such as blockchain, machine learning, and artificial intelligence could make run-of-the-mill clinical developers obsolete over time. Although these technologies can help with planning, monitoring, data management, and interpretation, these will never replace the personal interactions of experienced and empathetic professionals networking with all stakeholders in a clinical program.

About The Author:

Oscar Segurado, MD, Ph.D., is an executive veteran with extensive global leadership experience in translational science, clinical development, and global regulatory and medical affairs. He is the chief medical officer for ASC Therapeutics, a fast-growing biotechnology company focused on developing curative gene- and cell-based therapies for rare blood disorders. Segurado is the author and co-author of over 100 peer-reviewed publications, including Nature and Lancet, books, and medical articles, as well as a member of several scientific and medical societies, including his role as a Forbes Council Board Member. He also holds a tenured Professorship of Immunology at the University of Leon, Spain. He received his Ph.D. from the University of Wuerzburg, Germany, and MD from the University of Salamanca, Spain.